Therapeutic Development for Nonsense Mutations: The Final Frontier of CF

Emily’s Entourage (EE) announces its third scientific symposium, Therapeutic Development for Nonsense Mutations: The Final Frontier of CF, to be held September 24-25 at The Wistar Institute in Philadelphia, PA. The primary focus will be to accelerate breakthroughs and a cure for the final 10% of the cystic fibrosis (CF) population without a targeted therapy.

The Wistar Institute in Philadelphia, PA

With preeminent CF researchers and companies from around the world, this invitation-only meeting will be an intimate event harnessing collaboration, idea exchange, and creative problem solving to expedite the development of life-saving therapeutics for CF nonsense mutations. This area of therapeutic development is particularly important because game-changing CFTR modulating therapies are currently approved or in late-stage clinical development for roughly 90% of the CF population. Those with CF nonsense mutations represent the largest portion of the CF population without an approved or late-stage pipeline CFTR modulating therapy.

“At Emily’s Entourage, we are incredibly excited to bring together the best and brightest to collectively tackle nonsense mutations, which represents a major gap in therapeutic development for the CF community” – Emily Kramer-Golinkoff, EE Co-Founder

The symposium will commence with a welcome dinner the night prior to the meeting, where guests will be warmly received by EE Scientific Advisory Board Chair Kevin Foskett, PhD, Scientific Director Pete Haggie, PhD, and Emily. The objective of the dinner will be to mingle, network and articulate goals for the next day, where the hard, productive work will occur.

EE’s Key Opinion Leaders meeting in September 2015

On September 25, Emily will open with welcoming remarks, introducing people to Emily’s Entourage and its mission, putting a human face to the meeting’s goals and urgency. The meeting to follow will be divided into four key sessions, each consisting of short presentations and discussion. The four sessions are:

I. CFTR Modulators: Developing, adapting and testing modulators for truncated CFTRs

Chair: Pete Haggie, PhD (University of California, San Francisco, Emily’s Entourage)

II. CFTR Gene therapy: Progression to Phase I trials

Chair: Beverly Davidson, PhD (Children’s Hospital of Philadelphia, University of Pennsylvania, Spark Therapeutics, Talee Bio)

III. Nucleic acid therapies: Delivering mRNA, tRNA, and oligonucleotide-targeted approaches

Chairs: Phil Thomas (University of Texas, Southwestern) and Aliasger Salem (University of Iowa)

IV. Buying time: Alternative targets and approaches

Chair: Kevin Foskett, PhD (University of Pennsylvania, Emily’s Entourage)

List of presenters:

  • Alan Verkman, MD, PhD (University of California, San Francisco)
  • Neal Sharpe, PhD (Eloxx Pharmaceuticals)
  • Jeffrey Beekman, PhD (University Medical Center, Utrecht, Netherlands)
  • Steven M. Rowe, MD, MSPH (University of Alabama at Birmingham)
  • Paul McCray, MD (University of Iowa)
  • Patrick Harrison, PhD (University College Cork, Ireland)
  • Christopher Ahern, PhD (University of Iowa)
  • Lulu Huang, PhD  (Ionis Pharmaceuticals)
  • James Dahlman, PhD (Georgia Tech)
  • John Miller, PhD  (Proteostasis Therapeutics)
  • Robert Tarran, PhD (University of North Carolina at Chapel Hill, Spyryx Biosciences)
  • Oren Rosenberg, MD, PhD (University of California, San Francisco)
  • Barbara White, MD (Corbus Pharmaceuticals)
  • Martin D. Burke, MD, PhD (University of Illinois Urbana-Champaign)

Emily’s Entourage wants to thank the following sponsors whose support made this meeting possible:

Emily’s Entourage would like to express heartfelt gratitude to all the symposium speakers, attendees, and sponsors. We look forward to exciting new projects and collaborations resulting from the symposium that serve to expedite therapeutic development for CF nonsense mutations.

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